The U.S. Food and Drug Administration (FDA) has requested that UniQure conduct an additional study to validate the efficacy of its gene therapy for Huntington’s disease. An FDA official, speaking anonymously, revealed that the agency seeks a placebo-controlled trial for the treatment, which is administered directly to the brain. This requirement has led to controversy as UniQure argues that such a study would be unethical due to the need for patients to undergo lengthy general anesthesia. The FDA official, however, contended that UniQure may be aware of a previous trial’s inadequacies yet is attempting to manipulate comparison data to influence FDA perceptions.
This disagreement unfolds amid broader scrutiny of the FDA, with several recent drug application rejections prompting criticism from companies claiming inconsistent guidance from the agency. FDA Commissioner Marty Makary expressed criticism towards the therapy, suggesting it fell short of expectations without naming UniQure directly. In its defense, UniQure pointed out that its research relied on an external control database to illustrate the potential decline of patients without treatment, countering claims that the FDA had ever agreed to accept such comparisons as valid.
In response to recent allegations, UniQure expressed confidence in its data and described the FDA’s criticisms as irregular and misrepresentative of the submitted documents. Despite the ongoing tensions, UniQure’s stock saw an 18% increase, though it has plummeted by 56% throughout the year.
Why this story matters: The outcome may impact the future of gene therapies for Huntington’s disease and regulatory practices.
Key takeaway: The FDA insists on rigorous clinical trials to ensure drug efficacy, while UniQure defends its data as adequate for approval.
Opposing viewpoint: Critics argue that UniQure’s handling of trial data may mislead stakeholders about the therapy’s effectiveness.
